Scientists have discovered a new drug that rectifies a common mutation, which causes melanoma tumors. The drug only proved effective in phase 1 trials.

The study was conducted by Keith T. Flaherty, MD, of the Massachusetts General Hospital Cancer Center in Boston, and colleagues and was published in the New England Journal of Medicine on August 26.

The gene variant called the serine–threonine protein kinase B-RAF (BRAF) contains V600E, the mutation that leads to melanoma tumors.

The scientists, in the phase 1 trial, gave at least 240 mg of doses of PLX4032, or RG7204 to 55 patients suffering from melanoma. It was found that 10 of the 16 patients or 81% of the total patients reacted positively to the drug. One of the patients responded 100% to the mutation of BRAF. On the other hand, only 15% of the patients responded to chemotherapy and interleukin-2 (IL2), the standard treatments for melanoma.

In the second trial, 24 out of 32 patients responded somewhat to oral inhibitors to BRAF-mutated melanoma, while the two responded completely.

Jeffrey A. Sosman, MD, of Vanderbilt University in Nashville, Tenn and study’s co-author said, "These responses occur rapidly, in organs where we infrequently see tumor regression, and patients can actually feel better overnight with this therapy".

Dr. Alice Pentland, Chair of dermatology at the University of Rochester Medical Center, expressed his apprehensions regarding the findings, which cannot be generalized for all the masses.